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AU2001261063A1 - Dna sequences encoding dystrophin minigenes and methods of use thereof - Google Patents

Dna sequences encoding dystrophin minigenes and methods of use thereof

Info

Publication number
AU2001261063A1
AU2001261063A1 AU2001261063A AU6106301A AU2001261063A1 AU 2001261063 A1 AU2001261063 A1 AU 2001261063A1 AU 2001261063 A AU2001261063 A AU 2001261063A AU 6106301 A AU6106301 A AU 6106301A AU 2001261063 A1 AU2001261063 A1 AU 2001261063A1
Authority
AU
Australia
Prior art keywords
dystrophin
present
minigenes
provides
dystrophin minigenes
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
Application number
AU2001261063A
Inventor
Xiao Xiao
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Individual
Original Assignee
Individual
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Individual filed Critical Individual
Publication of AU2001261063A1 publication Critical patent/AU2001261063A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

The present invention provides a series of novel dystrophin minigenes that retain the essential biological functions. The expression of the dystrophin minigenes may be controlled by a regulatory element along with a small polyadenylation signal. The entire gene expression cassettes may be readily packaged into a viral vector, preferably an AAV vector. The present invention further defines the minimal functional domains of dystrophin and provides ways to optimize and create new versions of dystrophin minigenes. Finally, the present invention provides a method of treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
AU2001261063A 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof Abandoned AU2001261063A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US20077700P 2000-04-28 2000-04-28
US60/200,777 2000-04-28
PCT/US2001/013677 WO2001083695A2 (en) 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof

Publications (1)

Publication Number Publication Date
AU2001261063A1 true AU2001261063A1 (en) 2001-11-12

Family

ID=22743139

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001261063A Abandoned AU2001261063A1 (en) 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof

Country Status (9)

Country Link
US (2) US7001761B2 (en)
EP (1) EP1287125B1 (en)
CN (1) CN100422320C (en)
AT (1) ATE437944T1 (en)
AU (1) AU2001261063A1 (en)
CA (1) CA2407309C (en)
DE (1) DE60139394D1 (en)
ES (1) ES2330615T3 (en)
WO (1) WO2001083695A2 (en)

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WO2005118611A2 (en) * 2004-01-23 2005-12-15 The Trustees Of The University Of Pennsylvania Microutrophin and uses thereof
EP3808845A1 (en) 2004-06-28 2021-04-21 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
US7863017B2 (en) 2006-12-01 2011-01-04 Wisconsin Alumni Research Foundation TAT-utrophin as a protein therapy for dystrophinopathies
WO2008088895A2 (en) * 2007-01-18 2008-07-24 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma
US20080221027A1 (en) * 2007-03-09 2008-09-11 The Regents Of The University Of California Composition and Methods for the Treatment of Duchene Muscular Dystrophy
CA2942716C (en) 2007-07-12 2019-07-09 Biomarin Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
PT2203173E (en) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2249874A1 (en) 2008-02-08 2010-11-17 ProSensa Holding BV Methods and means for treating dna repeat instability associated genetic disorders
US20090280103A1 (en) * 2008-04-04 2009-11-12 Martin Flueck Regulation of muscle repair
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
BR122020021379B1 (en) 2008-10-24 2021-05-11 Sarepta Therapeutics, Inc. morpholino phosphorodiamidate oligomer, composition comprising the same and use of said oligomer to treat muscular dystrophy
JP2012524540A (en) 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Oligonucleotides containing inosine for treating DMD
DK2499249T3 (en) 2009-11-12 2018-12-03 Univ Western Australia ANTISENCE MOLECULES AND PROCEDURES FOR TREATING PATHOLOGIES
WO2011100505A2 (en) 2010-02-11 2011-08-18 Recombinetics, Inc. Methods and materials for producing transgenic artiodactyls
US10920242B2 (en) 2011-02-25 2021-02-16 Recombinetics, Inc. Non-meiotic allele introgression
CN112251436A (en) 2012-01-27 2021-01-22 比奥马林技术公司 RNA-regulated oligonucleotides with improved properties for the treatment of duchenne muscular dystrophy and becker muscular dystrophy
WO2014081855A1 (en) * 2012-11-20 2014-05-30 Universite De Montreal Methods and compositions for muscular dystrophies
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
WO2014144978A2 (en) 2013-03-15 2014-09-18 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
MA45477A (en) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital ADENOASSOCIATED VIRUS VECTOR VECTOR MICROARN-29 AND MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
EP3596112A2 (en) 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
WO2019078916A1 (en) 2017-10-18 2019-04-25 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
CA3099958A1 (en) * 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
AU2019290544B2 (en) 2018-06-18 2026-01-22 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
JP7069426B2 (en) * 2018-11-16 2022-05-17 アステラス製薬株式会社 Treatment of muscular dystrophy targeting the utrophin gene
KR20220016150A (en) 2019-05-30 2022-02-08 솔리드 바이오사이언시즈 인크. Recombinant Herpesvirales Vectors
EP3990030A1 (en) 2019-06-27 2022-05-04 Pfizer Inc. Methods of treating duchenne muscular dystrophy using aav mini-dystrophin gene therapy
CA3158281A1 (en) * 2019-10-25 2021-04-29 Audentes Therapeutics, Inc. Compositions and methods for treating glycogen storage disorders
WO2021089736A1 (en) 2019-11-06 2021-05-14 Association Institut De Myologie Combined therapy for muscular diseases
EP4121168A1 (en) * 2020-03-20 2023-01-25 University of Florida Research Foundation, Inc. Gene therapy for cockayne syndrome
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Also Published As

Publication number Publication date
EP1287125A2 (en) 2003-03-05
US20060073586A1 (en) 2006-04-06
EP1287125B1 (en) 2009-07-29
US7510867B2 (en) 2009-03-31
CA2407309C (en) 2011-08-02
EP1287125A4 (en) 2005-04-27
DE60139394D1 (en) 2009-09-10
US7001761B2 (en) 2006-02-21
WO2001083695A2 (en) 2001-11-08
US20030171312A1 (en) 2003-09-11
CN100422320C (en) 2008-10-01
WO2001083695A3 (en) 2002-03-07
ES2330615T3 (en) 2009-12-14
CA2407309A1 (en) 2001-11-08
ATE437944T1 (en) 2009-08-15
WO2001083695A9 (en) 2002-12-12
CN1439051A (en) 2003-08-27

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